Immune deficiencies, infection, and systemic immune disordersClinical outcome in children with chronic granulomatous disease managed conservatively or with hematopoietic stem cell transplantation
Section snippets
Methods
Children aged 16 years and less were eligible for this study because they are transitioned to adult care after this point. Potential participants were identified from the UK and Ireland CGD Registry, which was initially compiled in 2000 and was designed to identify all patients with CGD in the UK and Ireland.1 It was compiled by contacting consultants across the UK and Ireland and asking whether they had any patients with CGD. Once patients were enrolled, information was gathered by researchers
Results
Seventy-three living children were eligible for inclusion, of whom 59 (80%) were recruited. Five children who died before commencement of the study were also identified. Sixty-four (82%) patients were seen at the 2 national centers, and 14 children were identified from 5 other centers. Clinical information was abstracted for 62 children, 58 of the living children and 4 who died before commencement of the study. No records were available for data abstraction for 1 living and 1 deceased patient.
Discussion
This study reviewed the clinical course in children with CGD and those who have undergone transplantation. It demonstrates fewer infections and admissions to the hospital and improved growth in patients after transplantation compared with those who had not undergone transplantation. These data show that children in the UK with CGD have significant morbidity, with a serious infection once every 3.5 years; undergo a surgical procedure once every 4.8 years; and have an admission to the hospital
References (13)
- et al.
Clinical features, long term follow up and outcome of a large cohort of patients with chronic granulomatous disease: an Italian multicentre study
Clin Immunol
(2008) - et al.
Adherence to oral antidiabetic medications in the pediatric population with type 2 diabetes: a retrospective database analysis
Clin Ther
(2012) - et al.
Treatment of chronic granulomatous disease with myeloablative conditioning and an unmodified hemopoietic allograft: a survey of the European experience, 1985-2000
Blood
(2002) - et al.
Excellent survival after sibling or unrelated donor stem cell transplantation for chronic granulomatous disease
J Allergy Clin Immunol
(2012) - et al.
Special article: chronic granulomatous disease in the United Kingdom and Ireland: a comprehensive national patient based registry
Clin Exp Immunol
(2008) - et al.
Chronic granulomatous disease: the European experience
PLoS One
(2009)
Cited by (0)
This article presents independent research funded by the National Institute for Health Research (NIHR) under its Research for Patient Benefit (RfPB) Programme (grant reference no. PB-PG-0909-19060). The views expressed are those of the author(s) and not necessarily those of the National Health Service, the NIHR, or the Department of Health.
Disclosure of potential conflict of interest: T. Cole has received a grant from the National Institute for Health Research. The rest of the authors declare that they have no relevant conflicts of interest.